申请人:Ethris GmbH
公开号:EP3913054A2
公开(公告)日:2021-11-24
Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with a gene of the ATP-binding cassette (ABC) family, such as ABCA3.
本发明公开了编码肽、蛋白质、酶及其功能片段的多核苷酸。本发明公开的多核苷酸可有效地传递到器官,如肺,并在器官细胞内表达。本发明公开的多核苷酸可用于治疗与ATP结合盒(ABC)家族基因(如ABCA3)相关的疾病或病症。